Zhang Runhua, Liu Gaifen, Zhao Xingquan, Wang Yilong, Li Zixiao, Chen Guofang, Liu Bo, Ling Yun, Wang Yongjun, Li Shuya
Department of Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
China National Clinical Research Center for Neurological Diseases, Beijing, China.
Stroke Vasc Neurol. 2025 Apr 29;10(2):e003338. doi: 10.1136/svn-2024-003338.
GD-11, a novel brain cytoprotective drug, was designed to be actively taken up and transported across the blood-brain barrier via the glucose transporter. This study aimed to evaluate the safety and efficacy of GD-11 for improving the recovery of patients with acute ischaemic stroke (AIS).
A double-blind, randomised, placebo-controlled, phase 2 trial was conducted at 15 clinical sites in China. Patients aged 18-80 years with AIS within 48 hours were randomly assigned (1:1:1) to receive 160 mg GD-11, 80 mg GD-11 and placebo, two times a day for 10 days. The primary endpoint was a modified Rankin Scale (mRS) score of 0-1 at 90 days after treatment. The safety outcome was any adverse events within 90 days.
From 17 November 2022 to 22 March 2023, a total of 80 patients in the 160 mg GD-11 group, 79 patients in the 80 mg GD-11 group and 80 patients in the placebo group were included. The proportion of an mRS score of 0-1 at day 90 was 77.5% in the 160 mg GD-11 group, 72.2% in the 80 mg GD-11 group and 67.5% in the placebo group. Though no significant difference was found (p=0.3671), a numerically higher proportion was observed in the GD-11 group, especially in the 160 mg GD-11 group. The incidence of adverse events was similar across the three groups (p=0.1992).
GD-11 was safe and well-tolerated. A dosage of GD-11 160 mg two times a day was recommended for a large trial to investigate the efficacy.
新型脑保护药物GD-11旨在通过葡萄糖转运体被主动摄取并穿过血脑屏障。本研究旨在评估GD-11改善急性缺血性卒中（AIS）患者恢复情况的安全性和有效性。
在中国的15个临床地点进行了一项双盲、随机、安慰剂对照的2期试验。年龄在18-80岁、发病48小时内的AIS患者被随机分配（1:1:1）接受160mg GD-11、80mg GD-11和安慰剂治疗，每天两次，共10天。主要终点是治疗后90天时改良Rankin量表（mRS）评分为0-1分。安全性结局是90天内的任何不良事件。
从2022年11月17日至2023年3月22日，160mg GD-11组共纳入80例患者，80mg GD-11组纳入79例患者，安慰剂组纳入80例患者。160mg GD-11组在第90天时mRS评分为0-1分的比例为77.5%，80mg GD-11组为72.2%，安慰剂组为67.5%。虽然未发现显著差异（p=0.3671），但GD-11组的比例在数值上更高，尤其是160mg GD-11组。三组的不良事件发生率相似（p=0.1992）。
GD-11安全且耐受性良好。建议每天两次服用160mg GD-11进行大型试验以研究其疗效。
